Table 1.
Demographic and baseline clinical characteristics
| Characteristic | Study 201 | Study 103 | ||||||
|---|---|---|---|---|---|---|---|---|
| Cohort 1: | Cohort 2: | Cohort 3: | Cohort 4: | Overall, | Cohort 1: | Cohort 2: | Overall, | |
| 1000 mg q4w, | 1600 mg q6w, | 2400 mg q8w, | 5400 mg q12w, | 900 mg q4w, | 1800 mg q4w, | |||
| n = 6 | n = 6 | n = 7 | n = 7 | N = 26 | n = 6 | n = 7 | N = 13 | |
| Race, n (%) | ||||||||
| White | 5 (83.3) | 4 (66.7) | 3 (42.9) | 3 (42.9) | 15 (57.7) | 0 | 1 (14.3) | 1 (7.7) |
| Asian | 0 | 0 | 4 (57.1) | 3 (42.9) | 7 (26.9) | 6 (100) | 6 (85.7) | 12 (92.3) |
| Not reported | 1 (16.7) | 2 (33.3) | 0 | 0 | 3 (11.5) | 0 | 0 | 0 |
| Other | 0 | 0 | 0 | 1 (14.3) | 1 (3.8) | 0 | 0 | 0 |
| Sex, female, n (%) | 2 (33.3) | 1 (16.7) | 1 (14.3) | 2 (28.6) | 6 (23.1) | 2 (33.3) | 5 (71.4) | 7 (53.8) |
| Age at first infusion, mean (SD), y | 43.1 (14.57) | 48.6 (23.48) | 37.3 (14.03) | 48.5 (13.43) | 44.3 (16.33) | 41.1 (10.87) | 43.6 (13.48) | 42.4 (11.91) |
| Weight, mean (SD), kg | 73.3 (7.91) | 90.0 (15.58) | 73.6 (15.90) | 75.3 (13.72) | 77.8 (14.62) | 71.3 (11.06) | 64.7 (9.81) | 67.7 (10.51) |
| LDH, mean (SD), U/L | 1026.88 (547.843) | 1223.55 (149.693) | 2127.57 (815.875) | 2142.24 (366.511) | 1668.90 (724.341) | 1709.95 (582.118) | 1532.71 (355.050) | 1614.52 (461.172) |
| PNH granulocyte clone size, mean (SD), % | 85.75 (14.582) | 78.30 (24.323) | 79.42 (14.395)* | 91.06 (13.315) | 84.12 (16.914)† | 89.95 (6.981) | 91.10 (6.512) | 90.57 (6.470) |
| PNH type III erythrocyte clone size, mean (SD), % | 23.92 (20.002) | 28.25 (13.213) | 35.13 (18.331) | 32.39 (13.292) | 30.22 (16.016) | 27.08 (9.678) | 28.26 (12.813) | 27.72 (11.022) |
| Free hemoglobin, ‡ mean (SD), mg/L | 273.0 (172.29) | 234.5 (109.65) | 371.0 (278.16) | 507.0 (288.97) | 353.5 (242.05) | 237.2 (83.43) | 264.4 (109.05) | 251.8 (95.11) |
| Plasma haptoglobin,§ mean, g/L | <0.10 | <0.10 | <0.10 | <0.10 | <0.10 | <0.10 | <0.10 | <0.10 |
| Reticulocytes,|| mean (SD), ×1012/L | 0.1508 (0.06227) | 0.1685 (0.06071) | 0.2214 (0.03892) | 0.2259 (0.05690) | 0.1941 (0.06125) | 0.1970 (0.07080) | 0.1549 (0.05066) | 0.1743 (0.06205) |
| Platelets, mean (SD), ×109/L | 176.0 (141.45) | 184.3 (36.11) | 178.6 (92.45) | 215.3 (136.92) | 189.2 (105.28) | 246.2 (72.93) | 224.3 (129.21) | 234.4 (103.41) |
| Neutrophils, mean (SD), ×109/L | 3.282 (2.0649) | 1.783 (0.4950) | 2.291 (1.6252) | 2.401 (1.6134) | 2.432 (1.5622) | 2.885 (1.3742) | 2.724 (1.7062) | 2.798 (1.4998) |
| eGFR, mean (SD), mL/min/1.73 m2 | 88.58 (15.474) | 100.55 (23.351) | 119.73 (32.158) | 91.46 (21.372) | 100.50 (25.986) | 112.82 (21.691) | 115.81 (34.953) | 114.43 (28.448) |
| Patients receiving transfusions in 12 mo prior to ravulizumab dosing, n (%) | 0 | 2 (33.3) | 3 (42.9) | 2 (28.6) | 7 (26.9) | 2 (33.3) | 3 (42.9) | 5 (38.5) |
| FACIT-Fatigue score, mean (SD) | 32.0 (11.02) | 29.7 (17.32) | 28.7 (15.20) | 34.4 (10.08) | 31.2 (13.09) | 35.5 (10.03) | 25.4 (14.35) | 30.1 (13.12) |
| Disease history, n (%) | ||||||||
| Blood/lymphatic disorders | 2 (33.3) | 3 (50.0) | 3 (42.9) | 4 (57.1) | 12 (46.2) | 3 (50.0) | 3 (42.9) | 6 (46.2) |
| History of aplastic anemia | 2 (33.3) | 1 (16.7) | 0 | 2 (28.6) | 5 (19.2) | 1 (16.7) | 1 (14.3) | 2 (15.4) |
| Hepatobiliary disorders | 0 | 0 | 3 (42.9) | 1 (14.3) | 4 (15.4) | 2 (33.3) | 2 (28.6) | 4 (30.8) |
| Infections/infestations | 2 (33.3) | 3 (50.0) | 3 (42.9) | 0 | 8 (30.8) | 1 (16.7) | 2 (28.6) | 3 (23.1) |
| Renal/urinary disorders | 0 | 2 (33.3) | 1 (14.3) | 2 (28.6) | 5 (19.2) | 2 (33.3) | 1 (14.3) | 3 (23.1) |
| Cardiac disorders | 1 (16.7) | 1 (16.7) | 1 (14.3) | 0 | 3 (11.5) | 0 | 2 (28.6) | 2 (15.4) |
| Vascular disorders | 2 (33.3) | 2 (33.3) | 1 (14.3) | 4 (57.1) | 9 (34.6) | 1 (16.7) | 1 (14.3) | 2 (15.4) |
| History of MAVEs, n (%) | 0 | 1 (16.7) | 0 | 2 (28.6) | 3 (11.5) | 0 | 0 | 0 |
| History of antithrombotic medication use, n (%) | 1 (16.7) | 3 (50.0) | 3 (42.9) | 3 (42.9) | 10 (38.5) | 2 (33.3) | 1 (14.3) | 3 (23.1) |
| History of systemic corticosteroid use, n (%) | 2 (33.3) | 0 | 2 (28.6) | 3 (42.9) | 7 (26.9) | 3 (50.0) | 1 (14.3) | 4 (30.8) |
eGFR, estimated glomerular filtration rate; FACIT, Functional Analysis of Chronic Illness Therapy; MAVE, major adverse vascular event; ND, no data; q4w, every 4 weeks; q6w, every 6 weeks; q8w, every 8 weeks; q12w, every 12 weeks; SD, standard deviation.
*
n = 5.
†
n = 24.
‡
Upper limit of normal = 152 mg/L.
§
Reference range, 0.3 to 2.0 g/L; lower limit of detection, 0.1 g/L.
||
Reference range, 0.03 × 1012/L to 0.13 × 1012/L.